Approaches to advancing patient focussed outcomes assessment in - - PowerPoint PPT Presentation

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Approaches to advancing patient focussed outcomes assessment in - - PowerPoint PPT Presentation

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Approaches to advancing patient focussed outcomes assessment in clinical trials Jeremy Hobart MD, PhD Consultant Neurologist Professor of Clinical Neurology and Health Measurement EMA workshop on Multiple Sclerosis, London 17 TH October 2013

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Approaches to advancing patient focussed

  • utcomes assessment in clinical trials

Jeremy Hobart MD, PhD

Consultant Neurologist Professor of Clinical Neurology and Health Measurement EMA workshop on Multiple Sclerosis, London 17TH October 2013

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Workshop goals

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Overview

  • EMA draft guidance document – critique
  • Other guidance documents - relevance
  • Evidence / advances in measurement science
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Critique

  • Highlights importance of COA’s
  • Limited specifics on:

– COA selection – EMA expectations of selected COAs – Measurement (psychometric) method

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Relevance of other regulatory guidance

  • FDA also highlights importance of COA’s
  • More detailed specifics:

– COA selection – COA expectations – Methods

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Advances from measurement science

  • Application of new psychometric methods

– COA evaluation – COA modification – COA development

  • Conceptual clarity
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Item Response Theory

Rasch Measurement Theory

present 1900

Classical Test Theory

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What do new methods tell us?

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“Legacy” measures have critical limitations….that can be improved / fixed

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Clinically meaningful paths to scale improvement are clear….

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Standard analysis methods underestimate changes and difference…

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Traditional methods of evaluation change, and comparing ability to detect change, are misleading

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Legitimate individual–person level analysis

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Critical limitations of any statistical method

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“Disability”

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Importance...

Clinical Practice Implications Results Statistics Measurement

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Recommendations for advancing guidance

  • Focus on conceptual clarity of variables for

measurement

  • Encourage application of clinically meaningful modern

psychometric methods in COA development, evaluation, selection, and trial data analysis

  • Within-trial instrument evaluation
  • Consistency with other regulatory bodies